JP2017513500A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2017513500A5 JP2017513500A5 JP2016564019A JP2016564019A JP2017513500A5 JP 2017513500 A5 JP2017513500 A5 JP 2017513500A5 JP 2016564019 A JP2016564019 A JP 2016564019A JP 2016564019 A JP2016564019 A JP 2016564019A JP 2017513500 A5 JP2017513500 A5 JP 2017513500A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- raav
- protein
- pharmaceutical composition
- acid vector
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- 108020004707 nucleic acids Proteins 0.000 claims 27
- 102000039446 nucleic acids Human genes 0.000 claims 27
- 150000007523 nucleic acids Chemical class 0.000 claims 27
- 239000013598 vector Substances 0.000 claims 15
- 102000002233 Myelin-Oligodendrocyte Glycoprotein Human genes 0.000 claims 11
- 108010000123 Myelin-Oligodendrocyte Glycoprotein Proteins 0.000 claims 11
- 241000124008 Mammalia Species 0.000 claims 10
- 239000008194 pharmaceutical composition Substances 0.000 claims 10
- KISWVXRQTGLFGD-UHFFFAOYSA-N 2-[[2-[[6-amino-2-[[2-[[2-[[5-amino-2-[[2-[[1-[2-[[6-amino-2-[(2,5-diamino-5-oxopentanoyl)amino]hexanoyl]amino]-5-(diaminomethylideneamino)pentanoyl]pyrrolidine-2-carbonyl]amino]-3-hydroxypropanoyl]amino]-5-oxopentanoyl]amino]-5-(diaminomethylideneamino)p Chemical compound C1CCN(C(=O)C(CCCN=C(N)N)NC(=O)C(CCCCN)NC(=O)C(N)CCC(N)=O)C1C(=O)NC(CO)C(=O)NC(CCC(N)=O)C(=O)NC(CCCN=C(N)N)C(=O)NC(CO)C(=O)NC(CCCCN)C(=O)NC(C(=O)NC(CC(C)C)C(O)=O)CC1=CC=C(O)C=C1 KISWVXRQTGLFGD-UHFFFAOYSA-N 0.000 claims 6
- 208000023275 Autoimmune disease Diseases 0.000 claims 6
- 102000016202 Proteolipids Human genes 0.000 claims 6
- 108010010974 Proteolipids Proteins 0.000 claims 6
- 230000001225 therapeutic effect Effects 0.000 claims 5
- 241001465754 Metazoa Species 0.000 claims 4
- 239000003112 inhibitor Substances 0.000 claims 3
- 201000006417 multiple sclerosis Diseases 0.000 claims 3
- 239000002245 particle Substances 0.000 claims 3
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 108020003175 receptors Proteins 0.000 claims 3
- 102000005962 receptors Human genes 0.000 claims 3
- 208000024891 symptom Diseases 0.000 claims 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 102000008847 Serpin Human genes 0.000 claims 2
- 108050000761 Serpin Proteins 0.000 claims 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- -1 antibody Proteins 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 239000003102 growth factor Substances 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 2
- 239000003001 serine protease inhibitor Substances 0.000 claims 2
- QZAYGJVTTNCVMB-UHFFFAOYSA-N serotonin Chemical compound C1=C(O)C=C2C(CCN)=CNC2=C1 QZAYGJVTTNCVMB-UHFFFAOYSA-N 0.000 claims 2
- 229940124597 therapeutic agent Drugs 0.000 claims 2
- 102000004031 Carboxy-Lyases Human genes 0.000 claims 1
- 108090000489 Carboxy-Lyases Proteins 0.000 claims 1
- 102000014914 Carrier Proteins Human genes 0.000 claims 1
- 108010078791 Carrier Proteins Proteins 0.000 claims 1
- 102000053642 Catalytic RNA Human genes 0.000 claims 1
- 108090000994 Catalytic RNA Proteins 0.000 claims 1
- 101710112752 Cytotoxin Proteins 0.000 claims 1
- 208000016192 Demyelinating disease Diseases 0.000 claims 1
- 206010012305 Demyelination Diseases 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000003886 Glycoproteins Human genes 0.000 claims 1
- 108090000288 Glycoproteins Proteins 0.000 claims 1
- 101001115699 Homo sapiens Myelin-oligodendrocyte glycoprotein Proteins 0.000 claims 1
- 206010061218 Inflammation Diseases 0.000 claims 1
- 102000005755 Intercellular Signaling Peptides and Proteins Human genes 0.000 claims 1
- 108010070716 Intercellular Signaling Peptides and Proteins Proteins 0.000 claims 1
- 102000001617 Interferon Receptors Human genes 0.000 claims 1
- 102000014150 Interferons Human genes 0.000 claims 1
- 108010050904 Interferons Proteins 0.000 claims 1
- 102000015696 Interleukins Human genes 0.000 claims 1
- 108010063738 Interleukins Proteins 0.000 claims 1
- 102000047918 Myelin Basic Human genes 0.000 claims 1
- 102000006386 Myelin Proteins Human genes 0.000 claims 1
- 108010083674 Myelin Proteins Proteins 0.000 claims 1
- 101710107068 Myelin basic protein Proteins 0.000 claims 1
- 102000007339 Nerve Growth Factor Receptors Human genes 0.000 claims 1
- 108010032605 Nerve Growth Factor Receptors Proteins 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 108091005804 Peptidases Proteins 0.000 claims 1
- 108091093037 Peptide nucleic acid Proteins 0.000 claims 1
- 101710093543 Probable non-specific lipid-transfer protein Proteins 0.000 claims 1
- 239000004365 Protease Substances 0.000 claims 1
- 229940124158 Protease/peptidase inhibitor Drugs 0.000 claims 1
- 102000001253 Protein Kinase Human genes 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- 101710146873 Receptor-binding protein Proteins 0.000 claims 1
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 claims 1
- 108020004459 Small interfering RNA Proteins 0.000 claims 1
- 102000001742 Tumor Suppressor Proteins Human genes 0.000 claims 1
- 108010040002 Tumor Suppressor Proteins Proteins 0.000 claims 1
- 239000000556 agonist Substances 0.000 claims 1
- 239000005557 antagonist Substances 0.000 claims 1
- 230000002424 anti-apoptotic effect Effects 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 239000002246 antineoplastic agent Substances 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 102000003675 cytokine receptors Human genes 0.000 claims 1
- 108010057085 cytokine receptors Proteins 0.000 claims 1
- 229940127089 cytotoxic agent Drugs 0.000 claims 1
- 231100000599 cytotoxic agent Toxicity 0.000 claims 1
- 239000002619 cytotoxin Substances 0.000 claims 1
- 201000002491 encephalomyelitis Diseases 0.000 claims 1
- 239000012645 endogenous antigen Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 230000010437 erythropoiesis Effects 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 239000005556 hormone Substances 0.000 claims 1
- 229940088597 hormone Drugs 0.000 claims 1
- 108091008039 hormone receptors Proteins 0.000 claims 1
- 102000047972 human MOG Human genes 0.000 claims 1
- 230000006058 immune tolerance Effects 0.000 claims 1
- 208000027866 inflammatory disease Diseases 0.000 claims 1
- 230000004054 inflammatory process Effects 0.000 claims 1
- 229940079322 interferon Drugs 0.000 claims 1
- 229940043355 kinase inhibitor Drugs 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 210000005012 myelin Anatomy 0.000 claims 1
- 239000000137 peptide hydrolase inhibitor Substances 0.000 claims 1
- 108010011903 peptide receptors Proteins 0.000 claims 1
- 102000014187 peptide receptors Human genes 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108060006633 protein kinase Proteins 0.000 claims 1
- 239000003909 protein kinase inhibitor Substances 0.000 claims 1
- 239000013608 rAAV vector Substances 0.000 claims 1
- 210000003289 regulatory T cell Anatomy 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 229940076279 serotonin Drugs 0.000 claims 1
- 230000003612 virological effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019235107A JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461983924P | 2014-04-24 | 2014-04-24 | |
| US61/983,924 | 2014-04-24 | ||
| PCT/US2015/027598 WO2015164789A1 (en) | 2014-04-24 | 2015-04-24 | Aav-based gene therapy for multiple sclerosis |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019235107A Division JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017513500A JP2017513500A (ja) | 2017-06-01 |
| JP2017513500A5 true JP2017513500A5 (https=) | 2018-07-26 |
| JP6646282B2 JP6646282B2 (ja) | 2020-02-14 |
Family
ID=54333299
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016564019A Active JP6646282B2 (ja) | 2014-04-24 | 2015-04-24 | 多発性硬化症のためのａａｖ系遺伝子療法 |
| JP2019235107A Active JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
| JP2022031064A Pending JP2022081549A (ja) | 2014-04-24 | 2022-03-01 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019235107A Active JP7430898B2 (ja) | 2014-04-24 | 2019-12-25 | 多発性硬化症のためのａａｖ系遺伝子療法 |
| JP2022031064A Pending JP2022081549A (ja) | 2014-04-24 | 2022-03-01 | 多発性硬化症のためのａａｖ系遺伝子療法 |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US20170043036A1 (https=) |
| EP (2) | EP3133923B1 (https=) |
| JP (3) | JP6646282B2 (https=) |
| AU (2) | AU2015249328B2 (https=) |
| CA (1) | CA2945984C (https=) |
| CY (1) | CY1123312T1 (https=) |
| DK (1) | DK3133923T3 (https=) |
| ES (1) | ES2814280T3 (https=) |
| HR (1) | HRP20201342T1 (https=) |
| HU (1) | HUE050681T2 (https=) |
| LT (1) | LT3133923T (https=) |
| PL (1) | PL3133923T3 (https=) |
| PT (1) | PT3133923T (https=) |
| RS (1) | RS60816B1 (https=) |
| SI (1) | SI3133923T1 (https=) |
| SM (1) | SMT202000453T1 (https=) |
| WO (1) | WO2015164789A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7270946B2 (ja) * | 2017-08-08 | 2023-05-11 | 国立大学法人浜松医科大学 | 自己免疫疾患の治療又は予防剤 |
| BR112021003399A2 (pt) * | 2018-08-24 | 2021-05-18 | Spark Therapeutics, Inc. | sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso |
| CA3142487A1 (en) * | 2019-06-04 | 2020-12-10 | Thomas Jefferson University | Oligodendrocyte-derived extracellular vesicles for therapy of multiple sclerosis |
| WO2021211614A1 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Aav-based gene therapies for treatment of autoimmune diseases |
| WO2021211640A1 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Enhanced effects of gene-immunotherapy and immunosuppressants in multiple sclerosis |
| JP2023532923A (ja) * | 2020-06-30 | 2023-08-01 | チルドレンズ ナショナル メディカル センター | 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用 |
| WO2024081570A1 (en) * | 2022-10-10 | 2024-04-18 | Eli Lilly And Company | Methods and systems for using causal networks to develop models for evaluating biological processes |
| WO2024258925A1 (en) | 2023-06-12 | 2024-12-19 | Children's Hospital Medical Center | Aav-cftr vectors and methods of using same |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR960705038A (ko) * | 1993-09-03 | 1996-10-09 | 스테시 엘. 채닝 | 자기 면역 질환에 관련된 프로토콜에서 미엘린 올리고덴드로시트 당 단백질 및 그의 펩타이드 부분들의 용도(users of myelin oligodendrocyte glycoprotein and peptide portions thereof in protocols related to autoimmune desease) |
| AU5588298A (en) * | 1996-12-02 | 1998-06-29 | Cell Genesys, Inc. | Adeno-associated viral vector-mediated delivery of dna to cells of the liver |
| KR100449454B1 (ko) * | 2000-10-02 | 2004-09-21 | 이현철 | 단일사슬 인슐린 유도체의 유전자를 포함하는 당뇨병치료용 벡터 |
| EP1193272B1 (en) * | 2000-10-02 | 2004-06-30 | Yonsei University | Single-chain insulin analogs |
| CA2361462A1 (en) * | 2001-11-07 | 2003-05-08 | Katherine A. High | Induction of tolerance to a therapeutic polypeptide |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US20090304726A1 (en) * | 2006-02-15 | 2009-12-10 | Ramot At Tel Aviv University Ltd. | Viral display vehicles for treating multiple sclerosis |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| EP2364362B1 (en) * | 2008-11-12 | 2015-10-21 | Ospedale San Raffaele S.r.l. | Gene vector for inducing transgene-specific immune tolerance |
| JP6189754B2 (ja) * | 2011-03-04 | 2017-08-30 | イントレキソン コーポレーション | タンパク質を条件的に発現するベクター |
| JP2015502135A (ja) * | 2011-09-26 | 2015-01-22 | ウニヴェルジテート チューリッヒ プロレクトラート エムエヌヴェーＵｎｉｖｅｒｓｉｔａｅｔ Ｚｕｅｒｉｃｈ Ｐｒｏｒｅｋｔｏｒａｔ Ｍｎｗ | 多発性硬化症の治療のためのａｐｃ媒介寛容誘導 |
| US20160287685A1 (en) * | 2013-11-22 | 2016-10-06 | Amarna Holding B.V. | Method for restoring immune tolerance in vivo |
| WO2021211614A1 (en) * | 2020-04-14 | 2021-10-21 | University Of Florida Research Foundation, Incorporated | Aav-based gene therapies for treatment of autoimmune diseases |
-
2015
- 2015-04-24 EP EP15782332.9A patent/EP3133923B1/en active Active
- 2015-04-24 HU HUE15782332A patent/HUE050681T2/hu unknown
- 2015-04-24 PL PL15782332.9T patent/PL3133923T3/pl unknown
- 2015-04-24 RS RS20201008A patent/RS60816B1/sr unknown
- 2015-04-24 SM SM20200453T patent/SMT202000453T1/it unknown
- 2015-04-24 EP EP20167323.3A patent/EP3744177A1/en active Pending
- 2015-04-24 DK DK15782332.9T patent/DK3133923T3/da active
- 2015-04-24 AU AU2015249328A patent/AU2015249328B2/en not_active Ceased
- 2015-04-24 SI SI201531336T patent/SI3133923T1/sl unknown
- 2015-04-24 CA CA2945984A patent/CA2945984C/en active Active
- 2015-04-24 LT LTEP15782332.9T patent/LT3133923T/lt unknown
- 2015-04-24 WO PCT/US2015/027598 patent/WO2015164789A1/en not_active Ceased
- 2015-04-24 HR HRP20201342TT patent/HRP20201342T1/hr unknown
- 2015-04-24 JP JP2016564019A patent/JP6646282B2/ja active Active
- 2015-04-24 US US15/306,163 patent/US20170043036A1/en not_active Abandoned
- 2015-04-24 PT PT157823329T patent/PT3133923T/pt unknown
- 2015-04-24 ES ES15782332T patent/ES2814280T3/es active Active
-
2019
- 2019-04-17 AU AU2019202958A patent/AU2019202958B2/en not_active Ceased
- 2019-11-08 US US16/679,102 patent/US20200138978A1/en not_active Abandoned
- 2019-12-25 JP JP2019235107A patent/JP7430898B2/ja active Active
-
2020
- 2020-09-02 CY CY20201100820T patent/CY1123312T1/el unknown
-
2022
- 2022-03-01 JP JP2022031064A patent/JP2022081549A/ja active Pending
-
2023
- 2023-03-28 US US18/191,790 patent/US20230381342A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2017513500A5 (https=) | ||
| Zhang et al. | DKK3 ameliorates neuropathic pain via inhibiting ASK-1/JNK/p-38-mediated microglia polarization and neuroinflammation | |
| Jimenez et al. | FGF21 gene therapy as treatment for obesity and insulin resistance | |
| Eijkelkamp et al. | IL4-10 fusion protein is a novel drug to treat persistent inflammatory pain | |
| Fatoba et al. | Immunotherapies in Huntington's disease and α-Synucleinopathies | |
| Del Prete et al. | Adipokines as potential biomarkers in rheumatoid arthritis | |
| HRP20201342T1 (hr) | Genetska terapija za multiplu sklerozu bazirana na aav | |
| Miyagi et al. | Long-term aerobic exercise protects against cisplatin-induced nephrotoxicity by modulating the expression of IL-6 and HO-1 | |
| Naert et al. | Hematopoietic CC-chemokine receptor 2 (CCR2) competent cells are protective for the cognitive impairments and amyloid pathology in a transgenic mouse model of Alzheimer’s disease | |
| JP2020515643A5 (https=) | ||
| Onaga | Tachykinin: recent developments and novel roles in health and disease | |
| CN113677706B (zh) | 肝毒性的治疗 | |
| JP2017504568A5 (https=) | ||
| HRP20211559T1 (hr) | Rnk za liječenje autoimunih bolesti | |
| Song et al. | VIP enhances phagocytosis of fibrillar beta-amyloid by microglia and attenuates amyloid deposition in the brain of APP/PS1 mice | |
| Zheng et al. | Pivotal role of cerebral interleukin-23 during immunologic injury in delayed cerebral ischemia in mice | |
| Gonzalo-Gobernado et al. | Neuroprotective activity of peripherally administered liver growth factor in a rat model of Parkinson’s disease | |
| Vidinská et al. | Gradual phenotype development in Huntington disease transgenic minipig model at 24 months of age | |
| Kim et al. | Microglia in central control of metabolism | |
| EP3050570A1 (en) | Pharmaceutical composition consisting of a combination of G-CSF with GM-CSF | |
| Ikegami et al. | Monoclonal antibody against mature interleukin-18 ameliorates colitis in mice and improves epithelial barrier function | |
| Jian et al. | Role of growth factors in acute lung injury induced by paraquat in a rat model | |
| Moreno-Igoa et al. | Non-viral gene delivery of the GDNF, either alone or fused to the C-fragment of tetanus toxin protein, prolongs survival in a mouse ALS model | |
| RU2017104284A (ru) | Выделенные полипептиды cd44 и их применение | |
| US20170348393A1 (en) | Treatment of cns inflammatory disorders |